Targeting the genetic weaknesses in a specific type of childhood cancer

EXPLORE AND TARGET THE EPIGENETIC VULNERABILITY OF PAX3-FOXO1-DRIVEN RHABDOMYOSARCOMA

NIH-funded research St. Jude Children's Research Hospital · NIH-10878910

This study is looking at how certain genetic changes help a rare and aggressive childhood cancer called alveolar rhabdomyosarcoma grow, with the hope of finding new ways to treat it and improve outcomes for kids and teens facing this challenge.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionSt. Jude Children's Research Hospital NIH-funded
Lab location1 site (Memphis, United States)
Project IDNIH-10878910 on NIH RePORTER

What this research studies

This research focuses on understanding how certain genetic changes contribute to the development of a rare and aggressive childhood cancer called alveolar rhabdomyosarcoma. By investigating the role of specific proteins that drive tumor growth, the researchers aim to identify new treatment strategies that can block these proteins' functions. The study utilizes advanced techniques to analyze the genetic and epigenetic alterations in cancer cells, which may reveal new therapeutic targets. Ultimately, the goal is to improve treatment outcomes for affected children and adolescents.

Who could benefit from this research

Good fit: Ideal candidates for this research are children and adolescents diagnosed with alveolar rhabdomyosarcoma, particularly those with the PAX3-FOXO1 fusion gene.

Not a fit: Patients with other types of cancers or those who do not have the specific genetic markers associated with alveolar rhabdomyosarcoma may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could lead to more effective and targeted therapies for children with alveolar rhabdomyosarcoma, potentially improving survival rates and reducing long-term side effects.

How similar studies have performed: Previous research has shown promise in targeting genetic vulnerabilities in other cancers, suggesting that this approach may also be effective for alveolar rhabdomyosarcoma.

Where this research is happening

Memphis, United States

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Conditions Cancers
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.